Related Experiment Video
Updated: Nov 27, 2025

MicroRNA Amplification and Recognition through Locked-nucleic-acid In situ Hybridization as A Novel Detection and Quantification Method
Published on: October 7, 2025
The Therapeutic Potential of MicroRNAs in Cancer: Illusion or Opportunity?
Orazio Fortunato1, Marilena V Iorio2
1Tumor Genomics Unit, Department of Research, Fondazione IRCCS Istituto Nazionale dei Tumori, via Venezian 1, 20133 Milan, Italy.
Abstract:
The functional involvement of microRNAs in human neoplasia has raised in the last years an increasing interest in the scientific community toward the potential application in clinics as therapeutic tools. Indeed, the possibility to modulate their expression to re-establish a lost equilibrium and counteract tumor growth and dissemination, and/or to improve responsiveness to standard therapies, is promising and fascinating. However, several issues need to be taken into account such as factors related to miRNA stability in the blood, tissue penetration and potential off-target effects, which might affect safety, tolerability and efficacy of an miRNA-based therapy. Here we describe the most relevant challenges related to miRNA-based therapy, review the delivery strategies exploited to date and the on-going clinical trials.
Insights
MicroRNAs show promise for cancer therapy by modulating gene expression. However, challenges like stability, delivery, and off-target effects must be addressed for safe and effective clinical application.
Area of Science:
- Oncology
- Molecular Biology
- Biotechnology
Background:
- MicroRNAs (miRNAs) are increasingly recognized for their role in human neoplasia.
- The potential of modulating miRNA expression for cancer treatment is a significant area of research.
Purpose of the Study:
- To review the challenges associated with miRNA-based cancer therapy.
- To explore current delivery strategies for miRNA therapeutics.
- To summarize ongoing clinical trials in miRNA-based cancer therapy.
Main Methods:
- Literature review of miRNA function in neoplasia.
- Analysis of miRNA stability, tissue penetration, and off-target effects.
- Survey of existing miRNA delivery systems and clinical trial data.
Main Results:
- miRNA-based therapies offer potential for re-establishing cellular equilibrium and enhancing treatment response.
- Key challenges include miRNA stability in circulation, effective tissue penetration, and minimizing off-target effects.
- Various delivery strategies are being investigated to overcome these hurdles.
Conclusions:
- miRNA-based therapies hold promise for cancer treatment but require careful consideration of safety and efficacy.
- Addressing delivery and stability challenges is crucial for clinical translation.
- Ongoing clinical trials are evaluating the therapeutic potential of miRNAs in oncology.
Related Concept Videos
MicroRNAs
MicroRNAs
mTOR Signaling and Cancer Progression
The mTOR pathway or the...
lncRNA - Long Non-coding RNAs
Experimental RNAi
siRNA - Small Interfering RNAs
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...

